Koji Irisawa1,2, Masayuki Kaneko3, Mamoru Narukawa3. 1. Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1 Shirokane, Minato-ku, Tokyo, 108-8641, Japan. summerkoji0817@gmail.com. 2. Takeda Pharmaceutical Company Limited, Osaka, Japan. summerkoji0817@gmail.com. 3. Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1 Shirokane, Minato-ku, Tokyo, 108-8641, Japan.
Abstract
BACKGROUND: Accelerated approval (AA) is a program that grants approval to drugs based on clinical trial data for a surrogate endpoint or an intermediate clinical endpoint. Pharmaceutical companies are required to conduct a confirmatory trial to demonstrate true clinical benefit of the drug to obtain full approval (FA) for the AA. This study aimed at clarifying the points that should be considered by examining the characteristics of AA indications in all disease areas and the factors related to the status of conversion from AA to FA. METHODS: AA indications granted from January 1, 2000, to June 30, 2016, were investigated from the aspects of the characteristics of AAs and the status of conversion from AA to FA. RESULTS: Eighty-nine AAs were examined, of which 65 were converted to FA and 24 were not. A significant association was found between the FA status and period in which AA was granted, disease area, availability of IA data of a confirmatory trial for FA at the time of AA, and sales ranking of the company. CONCLUSIONS: To successfully convert from AA to FA, a development plan that focuses not only on AA but also on future FA needs to be considered and implemented from the early stage of development in line with the FDA guidance. In particular, for companies with insufficient experience in the development of AA indications and for products/indications without an established endpoint, more active discussion with the regulatory authorities from an early stage of development should be encouraged.
BACKGROUND: Accelerated approval (AA) is a program that grants approval to drugs based on clinical trial data for a surrogate endpoint or an intermediate clinical endpoint. Pharmaceutical companies are required to conduct a confirmatory trial to demonstrate true clinical benefit of the drug to obtain full approval (FA) for the AA. This study aimed at clarifying the points that should be considered by examining the characteristics of AA indications in all disease areas and the factors related to the status of conversion from AA to FA. METHODS: AA indications granted from January 1, 2000, to June 30, 2016, were investigated from the aspects of the characteristics of AAs and the status of conversion from AA to FA. RESULTS: Eighty-nine AAs were examined, of which 65 were converted to FA and 24 were not. A significant association was found between the FA status and period in which AA was granted, disease area, availability of IA data of a confirmatory trial for FA at the time of AA, and sales ranking of the company. CONCLUSIONS: To successfully convert from AA to FA, a development plan that focuses not only on AA but also on future FA needs to be considered and implemented from the early stage of development in line with the FDA guidance. In particular, for companies with insufficient experience in the development of AA indications and for products/indications without an established endpoint, more active discussion with the regulatory authorities from an early stage of development should be encouraged.
Keywords:
Accelerated approval; Drug development; FDA; Full approval; Regulatory science
Authors: Julia A Beaver; Lynn J Howie; Lorraine Pelosof; Tamy Kim; Jinzhong Liu; Kirsten B Goldberg; Rajeshwari Sridhara; Gideon M Blumenthal; Ann T Farrell; Patricia Keegan; Richard Pazdur; Paul G Kluetz Journal: JAMA Oncol Date: 2018-06-01 Impact factor: 31.777